Article this morning said there was hedge fund selling in a lot of the momo stocks yesterday as they try to get into the Alibaba IPO. BLUE is definitely a momo stock.
Looked up the Feuerstein-Ratain rule. It pertains to Phase 3 studies of cancer drugs (like NKTR-102 MBC). If the market cap of the stock is less than $300M four months before the release of the Phase 3 data, then the Phase 3 study will fail. Since 2000, the rule has 100% accuracy, it successfully predicted 36 study failures. Market cap above $300M, there is about 60% chance of success. NKTR market cap is well above $300M.
BLUE is an interesting speculation with explosive potential. BLUE is only 3% of my portfolio, if it drops more I may add a couple hundred shares. If BT and SCD fail, BLUE is going to the single digits, but it won't wipe me out. The science from this distance looks as good as any early clinical stage biotech I've seen.
FWIW, the data on patients-to-date was peer reviewed and presented at the 19th Annual Congress of the European Hematology Association (EHA) . The PI Dr. Marina Cavazzano is well respected in the field.
I googled "business phones". There was Vonage Business Services... in the paid ad section at the top. The actual search return for VBS didn't turn up until the fourth page.
Did I say it wasn't going to be approved?
What I do believe is that it will be years before meaningful royalties ( $100M/year) come from Movantik. And the label is going to play a role in it.
I'll add the details from the briefing document.
1) One died in car accident. I am certain this was due to Mova.
--Study 07 Patient E4073006 was a 54-year-old male in the naloxegol 12.5 mg group with diabetes. He was in a serious traffic accident on Day 146 (Day 60 of Study 07), after a “blackout”
attributed to hyperglycemia. The patient refused to be admitted to the hospital and left the hospital against medical advice. On Day 147, the patient was found dead. The autopsy listed the cause of death as ischemic heart disease secondary to coronary artery disease. This event was adjudicated as a CV death.
2) One died during an operation due to complications. This must have been because of Mova.
--Study 04 Patient E4068050 was a 73-year-old male in the naloxegol 12.5 mg group with multiple CV risk factors. He had a SAE of acute MI on Day 16 that led to surgery for aortic valve replacement and a coronary artery bypass graft, which was complicated by pneumonia, sepsis, and renal failure. The SAE of cardiac valve replacement on Day 19 resulted in the patient’s death on Day 49. This event was adjudicated as a CV death.
For example, BofA analyst Steve Byrne models peak adjusted Movantik sales of $380M in 2020. Let's say sales peak at $500M with an overly generous 20% royalty. That's $100M to NKTR in 2020. Less than that in the years leading up, minus the expenses for the NKTR share of the post approval study.
Now you know of something even close.
There were six patient CV deaths in the Movantik studies according to the FDA briefing documents, all of whom had taken Movantik at some point. The FDA documents supersede all of the AZN and NKTR press releases over the years and should be considered the definitive reference.
The Movantik label can matter too. OREX's Contrave got approved the other day with a 47 page label and a black box warning. The stock got hit hard.
But maybe the label won't matter much. The big thing for most people is the milestone money. There are concerns about how fast the market will ramp up even in the best case and what peak sales might be. Meaningful royalty money could take many years.
Clever Marty, about closed distribution and new doses/formulations.
Tiopronin is a very simple compound to make in a laboratory and it has been off-patent almost forever. But in order to get a another generic approved, you need to show equivalence to the existing drug. By going to closed distribution, RTRX effectively prohibits a potential competitor from obtaining Thiola to run an equivalence study. A standard Phase 3 for a competing tiopronin formulation would probably then need to have placebo as a control arm. But it would be unethical to deny cystinuria patients access to Thiola for an efficacy study. Going ahead with new doses and formulations of Thiola while discontinuing existing ones would present a moving target for any company trying to make its own generic formulation.
Besides, Marty says insurance companies, not patients, will be paying most of the bill, so what's the problem?
More good searches for those interested.
"Thiola, Retrophin, Martin Shkrell, Reddit, and More"
"A generic drug company (Retrophin) buys up the rights to a cheap treatment for a rare kidney disorder. And promptly jacks the price up 20x. A look at what they're up to"
On the Calendar of Events page, above the little calendar, there is a link labeled Past Events. That page has the MS presentation and other past presentations. The audio from MS is available, but presentation material hasn't been uploaded yet. There is presentation material from the previous events. I don't think much changed.